Pharmacists' Critical Role in Gene Therapy: Education, Collaboration, and Patient Care

In an interview with Pharmacy Times, Zahra Mahmoudjafari, PharmD, MBA, BCOP, FHOPA, clinical pharmacy manager in the Blood and Marrow Transplant Program in the Division of Hematologic Malignancies and Cellular Therapeutics at the University of Kansas Cancer Center in Kansas City, Kansas, discussed how gene therapy treatments represent a complex and evolving field. The discussion was based on the presentation “The Pharmacist’s Role in Gene Therapy Treatments,” highlighted at the American Pharmacists Association (APhA) Annual Meeting & Exposition.

During the discussion, Mahmoudjafari shares that gene therapy treatments are complex medical interventions spanning various disease states, where pharmacists play a pivotal role in patient education, clinical management, and interprofessional collaboration. She highlights that pharmacists are critical in managing both short-term and long-term toxicities, counseling patients and health care staff, and ensuring safe administration of treatments like cellular therapies and chimeric antigen receptor (CAR)-T cell interventions. The field requires extensive multidisciplinary teamwork, involving coordination between academic medical centers and community practices, with pharmacists navigating operational, clinical, and financial considerations. Mahmoudjafari emphasizes as gene therapies continue to evolve, pharmacists must stay updated through ongoing education, research, and collaboration to optimize patient outcomes and contribute to the advancement of these innovative treatments.

Pharmacy Times: How do pharmacists ensure the safe and effective administration of gene therapy treatments?

Zahra Mahmoudjafari: Gene therapy treatments are a little bit of a misnomer, because it's not just 1 type of treatment for 1 disease state. There are several different treatments that are available for various disease states. Those include not only patients with maybe some genetic impairments, but also patients with hematologic malignancies. In the setting of hematologic malignancies, with therapies like cellular therapies or CAR-T, pharmacists play a really important role with education for not only patients and their caregivers, but also for the health care staff. Making sure that the products are appropriately transitioned through a pharmacy and therapeutics committee, appropriately onboarded into the system, etc. Now for the other therapies that are considered more in vivo may not require that cell processing, manufacturing time, again, that is something where pharmacists are able to be a part of their patient's treatment that have gene therapy. Whether that be making sure the appropriate operational considerations have been taken into consideration as well. There's a lot that pharmacists are a part of, both clinically, operationally, and financially when it comes to gene therapies.

Pharmacy Times:What role do pharmacists play in education and counseling for gene therapy treatment?

Mahmoudjafari: They play a significant role, not only, like I said, education for patients and their caregivers, but also for other health care staff. These gene therapies are often incredibly complex, and so they really require the efforts of the multidisciplinary team. Pharmacists are well positioned to be able to advocate and incorporate our work within that space as well. The gene therapies are often very complex, and they do require the group, the multidisciplinary team overall. Again, pharmacists and their role with education and counseling can help not only understand the gene therapy itself, but sometimes these therapies have associated chemotherapy that's administered and some additional monitoring to be aware of, and that's both short term and long term. So again, pharmacists are very much involved and a part of the process.

Pharmacy Times: What is the pharmacist’s role in managing potential adverse effects and immune responses related to gene therapy?

Mahmoudjafari: The gene therapy, specifically cellular therapies, with therapies like CAR-T cell therapy or tumor infiltrating lymphocytes have not only acute toxicities, but also long-term toxicities. Pharmacists who are involved in maybe the short-term toxicities are helping manage symptoms like cytokine release syndrome or neurotoxicity, or maybe some complications from interleukin with the tumor infiltrating lymphocytes. We've learned a lot in terms of long-term safety monitoring as well. Many of these patients develop long term cytopenia and hypochromic anemia. So, making sure that patients are aware of those maybe long-term toxicities and patients are well managed and cared for even after that acute phase period.

Pharmacy Times: What are some of the logistical challenges in coordinating gene therapy treatments within a health care system and in coordination with community oncology pharmacy practices, as necessary?

Mahmoudjafari: There are a lot of steps here to keep into consideration. I always go back to the trifecta of operations, financial and clinical. Community practices serve a big role here in making sure that patients are appropriately referred largely to academic medical centers that offer these therapies. Most of these therapies, not all, but most of them do require to be administered under the care of a qualified treatment center, meaning that these centers have undergone rigorous training and protocols have been developed to be able to manage these patients appropriately. It is in partnership with our community practice colleagues to make sure that these patients are aware that these therapies are available, are referred to the academic practice in a timely manner. But then also, it's the responsibility of the academic medical centers then to transition those patients back to their community provider. Those providers are often closer to home, and we as the academic medical center should do a thorough job of educating our community practice colleagues also, again, with not only knowing what the indications are, but then the long-term toxicity, so that they can partner with us in their care.

Pharmacy Times: How can pharmacists contribute to pharmacovigilance and long-term monitoring for patients who have received gene therapy treatment?

Mahmoudjafari: This is a great question. This one is one that I think is still rapidly evolving. There's not a lot of standards published or a lot of available literature in this regard, because gene therapies are somewhat young in their utilization in a commercial setting. I think we're actively learning about that. Now even in the short time frame for which we've had cellular therapies, the first of which approved in 2017, we now have 8 years under our belt. We have learned a little more about long term monitoring. For example, some of the cellular therapy patients, there are recommendations to make sure these patients are revaccinated with some of their pediatric vaccines. Pharmacists can be aware of, educate to that, and make sure patients do receive their vaccinations again. There's a lot to be learned here. I think again, the pharmacists are well positioned to contribute to this space as well.

Pharmacy Times: What training and resources do you recommend for pharmacists stay up to date on gene therapy advancements?

Mahmoudjafari: The space is rapidly evolving. There isn't a single resource that's available. Many of the resources that are available are actively being published or in various journals and through various organizations. There are several different organizations that are specific to cell and gene therapy. If someone is interested in staying up in this space, it's either you learn about it through that specific disease state, or if you're someone like me who wants to have a comprehensive understanding of the landscape, it's being aware of what these other organizations are doing and the information that they have readily available and published. For me, I have subscribed to the various tables of contents for various journals, so that if something is published, I tend to stay up to date that way, I stay up to date through various social media channels. There are several different places you can go for this information. But a lot of it remains unpublished because we're actively learning about it as we practice. Maybe a call to action for folks to publish their experience or to collaborate, to be able to bring some of our experiences onto paper.

Pharmacy Times: How does reimbursement and insurance coverage impact access to gene therapy, and how do pharmacists assist with these challenges?

Mahmoudjafari: Reimbursement and insurance coverage for these, what we are now terming as ultra-high cost therapies are a significant challenge. These simply are just not available for patients who don't have any insurance. Then the other caveat that take into consideration is not just the treatment with the gene therapy, but the comprehensive package, or the workup to make patients ready for these therapies. Perhaps there's chemotherapy that needs to be administered. There's a corresponding admission that also goes with these therapies being given. Reimbursement insurance coverage are again another active area of change and updates. This is a space that for those that are very interested in the reimbursement element of things, it's a close area to watch. There's a lot going on and our payer colleagues are also struggling with the high cost of these therapies, and what does that look like? I try to think of that more comprehensively, not only the pharmacist and the health system being aware of patients being a candidate, making sure they have appropriate coverage. But then there's also pharmacists on the payer side who need to be aware of these therapies as well and make sure that these products are well evaluated and vetted within policies to make sure that they're covered.

Pharmacy Times:What interprofessional collaboration strategies can pharmacists use to optimize patient outcomes in gene therapy?

Mahmoudjafari: What I've really appreciated about being involved in this cell and gene therapy space for as long as I've been involved with it is the fact that you cannot, as a single department do this alone. It must be a collaborative effort amongst the multidisciplinary care team. That includes our typical players, with our physicians and our nurse nurses, but it also includes our apheresis cell processing team members, our individuals from finance, our revenue cycle team members, case managers and social workers. There is not a department that is not touched with these therapies. That brings its own set of challenges, but having a regularly, a routine schedule, time point for which to touch base, to make sure that all the I’s are dotted or Ts are crossed, are important in this studying. It's knowing who those colleagues are, that's probably half the battle is learning who those folks are in a large health system. But then once you learn who they are, you stick with them, and they get more well versed as well. It is a place that is an incredible place for teamwork and working together for the care of the patient. So, it's a lot of fun.